Rheumatology Research Group, School of Immunity and Infection, College of Medical and Dental Sciences, University of Birmingham, Edgbaston, B15 2TT, UK.
Expert Rev Clin Immunol. 2010 Nov;6(6):831-41. doi: 10.1586/eci.10.66.
The cryopyrin-associated periodic syndromes (CAPS) are a group of rare inherited inflammatory diseases associated with overproduction of IL-1β. Canakinumab, developed by Novartis AG (Basel, Switzerland), is an intravenously or subcutaneously administered, fully human monoclonal antibody that neutralizes the bioactivity of human IL-1β. Canakinumab has promising clinical safety and pharmacokinetic properties, and has demonstrated potential for the treatment of CAPS. Canakinumab was recently granted EU orphan drug status for systemic-onset juvenile idiopathic arthritis, and early clinical trials have established that administration of canakinumab every 2 weeks is both safe and effective. Subcutaneous canakinumab (approved formulation) offers some advantages over the existing IL-1β-blocking treatment, anakinra, which must be injected daily and is often not well tolerated by patients. The long-term safety of all targeted anti-IL-1 therapies in CAPS remains an unanswered question owing to the relatively short clinical experience with these agents; as canakinumab produces sustained IL-1 suppression, vigilance is necessary to diagnose the development of adverse events, especially any associated infections.
cryopyrin 相关周期性综合征（CAPS）是一组罕见的遗传性炎症性疾病，与白细胞介素-1β（IL-1β）的过度产生有关。卡那单抗（由瑞士巴塞尔诺华制药公司研发）是一种静脉注射或皮下注射的、全人源单克隆抗体，可中和人白细胞介素-1β 的生物活性。卡那单抗具有良好的临床安全性和药代动力学特性，具有治疗 CAPS 的潜力。卡那单抗最近被授予用于全身型幼年特发性关节炎的欧盟孤儿药地位，早期临床试验已经证实，每两周给药一次卡那单抗既安全又有效。与现有的 IL-1β 阻断治疗药物阿那白滞素（anakinra）相比，皮下注射用卡那单抗（批准的制剂）具有一些优势，阿那白滞素必须每天注射，且往往不能被患者很好地耐受。由于这些药物的临床经验相对较短，所有靶向抗 IL-1 治疗在 CAPS 中的长期安全性仍然是一个悬而未决的问题；由于卡那单抗可产生持续的 IL-1 抑制作用，因此需要保持警惕以诊断不良事件的发生，尤其是任何相关感染。
